Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Inclusion Criteria: * Genetically confirmed diagnosis of Familial Dysautonomia. * Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database. * One or more autonomic crises during the last year. * Age above 18 years. * The patient has a responsible caretaker to communicate with the medical providers. * Provision of signed and dated informed consent form from the patient and responsible caregiver. * Able to state willingness to comply with all study procedures and availability for the duration of the study * For males and females of reproductive potential: use condoms for contraception if sexually active. Exclusion Criteria: * At the consideration of the principal investigator, the caregiver cannot fully understand the protocol or communicate during the crisis with the Center. * The patient during the crisis, before taking the medication, has any of the following: * a. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency. * b. Respiratory rate \>20 breaths per minute. * c. Supine blood pressure ≤ 90/60mmHg * d. Febrile illness with temperature \>100.3 F. * e. Serological signs of infection (WBC count \>10 g/dL, or CRP \>10 mg/L or ESR\>20, or above their steady historical baseline levels) in recent (less than one month) studies. * The patient is a female and has a positive pregnancy test. *…